Regulatory environment too tough for orphan drugs?

The number of marketing authorisations granted to orphan drugs has been falling, and Boehringer Ingelheim’s respiratory medical manager puts this down to orphan drug fims having a difficult time in the current regulatory environment. “That decline shows how difficult it is for companies to show the value of medicines and develop them for rare diseases,” said Dr Aruni Mulgirigama.

Difficulties include:

Developing a clinical trial that demonstrates efficacy against a rare disease when current regulation demands data from thousands of patients

Do you think the regulatory environment is too touch for orphan drugs? Does the standards need to change?

Recently, the European Court of Justice threw out a decision by the European Commission (EC) not to approve Orphacol. Because the liver indication was so rare, CTRS, the drug’s manufacturer, had instead submitted references to scientific literature to back up its application rather than the results of clinical trials.

The European Court ruled that ‘well-established medicinal use’ had been demonstrated by CTRS, and accepted that the rareness of the disorders and the risk of serious liver damage associated with a clinical trial meant that CTRS can not carry out a controlled study of the drug’s efficacy. The trial ignited discussion on the complications of regulating orphan drugs where there is a relative lack of clinical information. Read more about this story >